# Precision Medicine for Inflammatory Treatment for Alzheimer's Disease in Down Syndrome

> **NIH NIH R01** · UNIVERSITY OF SOUTHERN CALIFORNIA · 2024 · $623,408

## Abstract

PROJECT SUMMARY/ABSTRACT
Down syndrome (DS) is the most common genetic cause of intellectual disability, with virtually all DS individuals
having AD by age 60. Despite exciting advances over the past 20 years in our understanding of DS and how
genetics confer susceptibility for AD, there have been few clinical trials to treat AD in the DS population (DS-AD)
and currently no means to clinically impact disease progression. We hypothesize that the biological makeup of
DS-AD is as heterogeneous as the sporadic AD population and that identifying specific subsets of DS-AD for
particular treatments will yield meaningful therapeutic responses. We propose to test a validated biomarker for
sporadic AD, our established proinflammatory endophenotype analysis. This analysis will allow for an
understanding of differences in immune signaling between DS-AD individuals and how this may impact anti-
inflammatory effects on AD biomarkers. For this study we will leverage biobanked samples from the previously
completed Phase 3 clinical trial, “Vitamin E in Aged Persons with Down Syndrome (NCT00056329).” For our
proposed work we will employ high throughput proteomics on native plasma and exosome subpopulations to
validate new techniques and develop a framework for designing better therapeutic trials for DS-AD

## Key facts

- **NIH application ID:** 11019654
- **Project number:** 4R01AG073979-02
- **Recipient organization:** UNIVERSITY OF SOUTHERN CALIFORNIA
- **Principal Investigator:** Michael S Rafii
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2024
- **Award amount:** $623,408
- **Award type:** 4N
- **Project period:** 2021-09-30 → 2026-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/11019654

## Citation

> US National Institutes of Health, RePORTER application 11019654, Precision Medicine for Inflammatory Treatment for Alzheimer's Disease in Down Syndrome (4R01AG073979-02). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/11019654. Licensed CC0.

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